BR112018011975A2 - composições úteis no tratamento de atrofia muscular espinhal - Google Patents

composições úteis no tratamento de atrofia muscular espinhal

Info

Publication number
BR112018011975A2
BR112018011975A2 BR112018011975A BR112018011975A BR112018011975A2 BR 112018011975 A2 BR112018011975 A2 BR 112018011975A2 BR 112018011975 A BR112018011975 A BR 112018011975A BR 112018011975 A BR112018011975 A BR 112018011975A BR 112018011975 A2 BR112018011975 A2 BR 112018011975A2
Authority
BR
Brazil
Prior art keywords
treatment
muscular atrophy
spinal muscular
compositions
compositions useful
Prior art date
Application number
BR112018011975A
Other languages
English (en)
Inventor
Hinderer Christain
M Wilson James
Original Assignee
Univ Pennsylvania
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Univ Pennsylvania filed Critical Univ Pennsylvania
Publication of BR112018011975A2 publication Critical patent/BR112018011975A2/pt

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Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/60—Vectors comprising as targeting moiety peptide derived from defined protein from viruses
    • C12N2810/6027—Vectors comprising as targeting moiety peptide derived from defined protein from viruses ssDNA viruses

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Molecular Biology (AREA)
  • Public Health (AREA)
  • Biochemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biotechnology (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • Biophysics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Toxicology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Immunology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Neurosurgery (AREA)
  • Psychology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

a presente invenção refere-se a composições e métodos úteis no tratamento de atrofia muscular espinhal. as composições compreendem um vetor viral adenoassociado recombinante que contém um capsídeo de aav, por exemplo, capsídeo de aavrh.10, e as sequências de ácido nucleico que codificam uma proteína de smn funcional. os métodos envolvem a administração destas composições em humanos em necessidade dos mesmos.
BR112018011975A 2015-12-14 2016-12-14 composições úteis no tratamento de atrofia muscular espinhal BR112018011975A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562267012P 2015-12-14 2015-12-14
PCT/US2016/066669 WO2017106354A1 (en) 2015-12-14 2016-12-14 Adeno-associated viral vectors useful in treatment of spinal muscular atropy

Publications (1)

Publication Number Publication Date
BR112018011975A2 true BR112018011975A2 (pt) 2018-12-11

Family

ID=57737990

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112018011975A BR112018011975A2 (pt) 2015-12-14 2016-12-14 composições úteis no tratamento de atrofia muscular espinhal

Country Status (13)

Country Link
US (2) US20180353624A1 (pt)
EP (1) EP3394270A1 (pt)
JP (1) JP7082050B2 (pt)
KR (1) KR20180086266A (pt)
CN (1) CN109072254A (pt)
AU (1) AU2016370630B2 (pt)
BR (1) BR112018011975A2 (pt)
CA (1) CA3008280A1 (pt)
IL (1) IL259877A (pt)
MA (1) MA44119A (pt)
MX (1) MX2018007234A (pt)
WO (1) WO2017106354A1 (pt)
ZA (1) ZA201803956B (pt)

Families Citing this family (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG10202100632WA (en) * 2016-07-26 2021-03-30 Univ Cornell Gene therapy for the treatment of aldehyde dehydrogenase deficiency
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
MD3589730T2 (ro) 2017-02-28 2024-04-30 Univ Pennsylvania Vector de virus adeno-asociat (AAV) de cladă F și utilizările acestuia
WO2019040586A1 (en) * 2017-08-25 2019-02-28 Ovid Therapeutics Inc. RECOMBINANT ADENO-ASSOCIATE VECTORS
WO2020037161A1 (en) * 2018-08-15 2020-02-20 Biogen Ma Inc. Combination therapy for spinal muscular atrophy
CA3122319A1 (en) * 2018-12-21 2020-06-25 Genethon Expression cassettes for gene therapy vectors
US20220280548A1 (en) * 2019-08-15 2022-09-08 Biogen Ma Inc. Combination therapy for spinal muscular atrophy
CN112011571A (zh) * 2020-04-26 2020-12-01 辉大(上海)生物科技有限公司 一种用于治疗脊髓性肌萎缩的基因治疗药物
WO2021219762A1 (en) * 2020-04-28 2021-11-04 Genethon Use of a synthetic aav capsid for gene therapy of muscle and central nervous system disorders
US20230304034A1 (en) 2020-05-12 2023-09-28 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
CN113755524B (zh) * 2020-06-02 2023-11-03 舒泰神（北京）生物制药股份有限公司 用于治疗脊髓性肌萎缩的腺相关病毒载体及其用途
RU2742837C1 (ru) * 2020-06-02 2021-02-11 Общество С Ограниченной Ответственностью "Анабион" Кодон-оптимизированная нуклеиновая кислота, которая кодирует белок SMN1, и ее применение
BR112023015303A2 (pt) 2021-02-01 2023-11-14 Regenxbio Inc Método para tratar doença cln2 devido a deficiência de tpp1 em um sujeito
CN112852882A (zh) * 2021-02-04 2021-05-28 中吉智药(南京)生物技术有限公司 一种杆状病毒感染昆虫细胞生产aav基因药物的系统及方法
IL312487A (en) * 2021-11-09 2024-07-01 Asimov Inc Stable production systems for adeno-associated virus vector production
WO2023087019A2 (en) 2021-11-15 2023-05-19 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
WO2023219394A1 (ko) * 2022-05-10 2023-11-16 서울대학교산학협력단 인간 smn1 단백질 변이체 및 이의 용도

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SI2292780T1 (sl) * 2003-09-30 2017-12-29 The Trustees Of The University Of Pennsylvania Genotipske skupine, zaporedja, vektorji, ki vsebujejo le-te z adenovirusi povezanega virusa (aav) in njihova uporaba
CA2877428A1 (en) * 2012-06-21 2013-12-27 Association Institut De Myologie Widespread gene delivery of gene therapy vectors
BR112015027336A2 (pt) * 2013-05-01 2017-09-26 Genzyme Corp composições e processos para tratamento de atrofia muscular espinhal
BR112016008832A2 (pt) * 2013-10-22 2017-10-03 Shire Human Genetic Therapies Distribuição de mrna no snc e suas utilizações
GB201403684D0 (en) * 2014-03-03 2014-04-16 King S College London Vector

Also Published As

Publication number Publication date
CA3008280A1 (en) 2017-06-22
AU2016370630A1 (en) 2018-06-28
US20220265861A1 (en) 2022-08-25
IL259877A (en) 2018-07-31
CN109072254A (zh) 2018-12-21
JP7082050B2 (ja) 2022-06-07
KR20180086266A (ko) 2018-07-30
MA44119A (fr) 2018-10-31
JP2018537984A (ja) 2018-12-27
EP3394270A1 (en) 2018-10-31
MX2018007234A (es) 2018-11-09
WO2017106354A1 (en) 2017-06-22
US20180353624A1 (en) 2018-12-13
ZA201803956B (en) 2019-04-24
AU2016370630B2 (en) 2023-04-13

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B07D Technical examination (opinion) related to article 229 of industrial property law [chapter 7.4 patent gazette]

Free format text: DE ACORDO COM O ARTIGO 229-C DA LEI NO 10196/2001, QUE MODIFICOU A LEI NO 9279/96, A CONCESSAO DA PATENTE ESTA CONDICIONADA A ANUENCIA PREVIA DA ANVISA. CONSIDERANDO A APROVACAO DOS TERMOS DO PARECER NO 337/PGF/EA/2010, BEM COMO A PORTARIA INTERMINISTERIAL NO 1065 DE 24/05/2012, ENCAMINHA-SE O PRESENTE PEDIDO PARA AS PROVIDENCIAS CABIVEIS.

B07E Notification of approval relating to section 229 industrial property law [chapter 7.5 patent gazette]
B06U Preliminary requirement: requests with searches performed by other patent offices: procedure suspended [chapter 6.21 patent gazette]
B350 Update of information on the portal [chapter 15.35 patent gazette]
B07A Application suspended after technical examination (opinion) [chapter 7.1 patent gazette]
B09B Patent application refused [chapter 9.2 patent gazette]
B09B Patent application refused [chapter 9.2 patent gazette]

Free format text: MANTIDO O INDEFERIMENTO UMA VEZ QUE NAO FOI APRESENTADO RECURSO DENTRO DO PRAZO LEGAL.MANTIDO O INDEFERIMENTO UMA VEZ QUE NAO FOI APRESENTADO RECURSO DENTRO DO PRAZO LEGAL.